Phase 3Data readout

NGN-401

Rett syndrome

NGNENeurogene Inc

Expected catalystDecember 31, 2027Company guidance: Dec 31, 2027

Catalyst overview

Event details

Drug or asset: NGN-401
Development stage: Phase 3
Event type: Data readout
Indication: Rett syndrome

Research signalsHigh management interest

Research summary

What is being tracked?

This catalyst tracks a Phase 3 data readout for NGN-401 in Rett syndrome. The expected timing is based on the latest available company guidance and may change.

Development notes

11/11/24- Reported interim data from first 4 participants. 11/6/25- First patient dosed in registrational trial. 11/12/25- Interim data reported & presentation at Stifel'25. 11/13/25- Complete enrollment in 3-6mths. 01/12/26:- Embolden registrational trial dosing completes Q2 2026. Ph1/2 interim data expected Mid-2026. 02/26/26:- FDA granted Breakthrough Therapy to NGN-401. Embolden dosing on track Q2 2026; more Ph1/2 data Mid-2026. 03/24/26:- Embolden fully enrolled; >50% dosed. Ph1/2 updated data and PPQ start expected Mid-2026. 04/28/26:- ASGCT poster May 12, 2026 highlights ICV delivery and Ph1/2 interim data. 05/12/26:- ASGCT poster: Ph1/2 dosing completed; pediatric cohort showed milestones; well-tolerated to 10/30/25. Embolden ~90% dosed; dosing completion expected Q2 2026; mid-2026 Ph1/2 data and PPQ initiation reaffirmed. 06/08/26:- Embolden dosing completed (25 patients). Updated Ph1/2 data mid-2026; Embolden topline expected H2 2027. 06/29/26:- Updated Ph1/2 data cut June 16, 2026. Embolden topline still expected H2 2027. 08/12/26:- Ph1/2 data were positive. PPQ runs expected end-2026. Embolden topline H2 2027.

Verification

Primary source

Confirm timing and program details against the company announcement or filing.

Open source

What is being tracked?

This catalyst tracks a Phase 3 data readout for NGN-401 in Rett syndrome. The expected timing is based on the latest available company guidance and may change.